A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria)

Merck KGaA0 个研究点目标入组 30 人开始时间: 2013年7月26日最近更新: 2015年4月7日

适应症

试验速览

阶段: 不适用
状态: 进行中（未招募）
发起方: Merck KGaA
入组人数: 30

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional clinical trial of medicinal product

入排标准

性别: All

入选标准

•Written informed consent given before any trial-related activities are carried out.
•Women or men with documented PKU diagnosed by at least two Phe levels = 400 µmol/L.
•Willingness to use a highly effective method of
•contraception is required during the study and follow-up
•periods. For women of childbearing potential: a negative urine pregnancy test is required at the end of screening.
•Aged = 18 to 29 years, inclusive.
•Mean blood Phe levels 600 to 1000 µmol/L during 12 months preceding inclusion in the study. The mean should be calculated from at least 3 blood Phe values over the last 12 months. Screening blood Phe level can be one of these values. There should be at least one value dated between Month -12 and -6 before screening and at least one value dated between Month -6 and screening.
•An intelligence quotient (IQ) score = 85, assessed a maximum of 2 years before screening with an age appropriate Wechsler scale. If no IQ test result is available, IQ testing must be performed as part of screening using an age-appropriate Wechsler scale before the subject can be included.
•Subjects willing to comply with all study procedures, including willingness to continue current dietary recommendations during the whole trial duration.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 30
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

排除标准

•Subjects with tetrahydrobiopterin (BH4) deficiency.
•Previous exposure to sapropterin or BH4 for > 30 days (or exposure to sapropterin or BH4 for = 30 days but within the previous 6 months prior to screening visit).
•Subjects who, according to the Investigator, will not be able to comply with study procedures and computerized neuropsychological testing.
•Any significant illness which, according to the Investigator, might preclude participation in the study (including neurological disease, cardio-vascular disease, history of seizure, predisposition to convulsions, renal or hepatic insufficiency, and active malignancy).
•Any significant illness, medication or substance abuse which, according to the Investigator, might affect cognitive function and cognitive testing (e.g., significant visual or motor impairment, history of major head trauma, history of stroke, alcoholism, drug dependency, psychological disorder requiring chronic use of psychotropic medications such as anxiolytics, antidepressants, antipsychotic medication, mood stabilizers, and hypnotics).
•Concomitant forbidden medication as described in the Kuvan® Summary of Product Characteristics, namely, inhibitors of dihydrofolate reductase (e.g., methotrexate, trimethoprim), medications that are known to affect nitric oxide synthesis (e.g., glyceryl trinitrate, isosorbide dinitrate, sodium nitroprusside, molsidomin, phosphodiesterase type 5 inhibitors, and minoxidil), and levodopa, as it may cause increased excitability and irritability.
•Known hypersensitivity to sapropterin or any ingredients in the product’s formulation, or to other approved or non-approved formulations of BH4.
•Subjects who have undergone cognitive neuropsychological testing similar to that to be performed as part of this trial with the following time limits: tasks with limited practice effect performed in the last 6 months, and tasks with important practice effect (such as tasks involving development of strategies) performed in the year preceding inclusion in the trial. Whether a task falls into one or the other category is left to Investigator judgment.
•Female subjects who are pregnant or in the lactation period.
•Subjects currently participating in another clinical trial or who participated in a previous clinical trial within 30 days prior to screening.
•Legal incapacity or limited legal capacity.

研究者

发起方

Merck KGaA

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A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine.MedDRA version: 16.0Level: LLTClassification code 10034873Term: Phenylketonuria (PKU)System Organ Class: 100000004850Phenylketonuria

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